Corneal Endothelial Dystrophy Type 2

SEATTLE & CAMBRIDGE, Mass. & TOKYO--( BUSINESS WIRE )-- Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that it will present clinical data on its allogeneic cell therapy drug candidate, AURN001, at the 42 nd Congress of the European Society of Cataract and Refractive Surgeons (ESCRS 2024) to be held September 6-10, 2024 in Barcelona, Spain. Aurion Biotech also will present and participate in several panel discussions during the Ophthalmology Futures European Forum , which takes place September 5 th in Barcelona. “We are thrilled to share clinical findings, as well as clinical and business milestones in multiple sessions at ESCRS and the Ophthalmology Futures European Forum,” said Dr. Michael Goldstein, president and chief medical Officer. “In particular, we believe that efficacy and safety data from the Escalón clinical trial provide additional insights into how our combination drug candidate may have a positive impact on the lives of patients suffering from corneal endothelial dysfunction.” The schedule of presentations is as follows: Ophthalmology Futures European Forum Will Novel Dry Eye Therapies Really Make a Difference? Thursday, September 5, 2024 1:15 p.m. – 1:45 p.m. CET The Esferic, Barcelona Panelist: Michael Goldstein, MD, MBA, President and Chief Medical Officer Cell, Gene and other Novel Corneal Therapies: Outlook for the Second Quarter of the 21 st Century Thursday, September 5, 2024 1:45 p.m. – 2:15 p.m. CET The Esferic, Barcelona Panelist: Greg Kunst, Chief Executive Officer Transformational Cell Therapy for Corneal Endothelial Disease Thursday, September 5, 2024 2:30 p.m. – 3:15 p.m. CET The Esferic, Barcelona Presenter: Greg Kunst, Chief Executive Officer Patient- vs. Provider-Centric Biotech & MedTech Innovation Thursday, September 5, 2024 6:45 p.m. – 7:15 p.m. CET The Esferic, Barcelona Panelist: Eris Jordan, OD, Vice President, Global Clinical & Medical Affairs ESCRS iNovation Day Transformational Cell Therapy for Corneal Endothelial Disease Friday, September 6, 2024 11:26 a.m. – 12:23 p.m. CET Fira Gran Via, Barcelona Presenter: Greg Kunst, Chief Executive Officer ESCRS Human Corneal Endothelial Cell Transplantation Combined with Cataract Extraction and PCIOL in Subject with Corneal Edema Sunday September 8, 2024 1:15 p.m. – 1:45 p.m. CET Pod 1 (between Exhibition Halls), Fira Gran Via, Barcelona Presenter: Michael Goldstein, MD, MBA, President and Chief Medical Officer Escalón: 12-Month Safety and Efficacy Clinical Trial of Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to Endothelial Dysfunction Monday, September 9, 2024 5:30 p.m. – 5:36 p.m. CET Free paper podium 2, Fira Gran Via, Barcelona Presenter: Michael Goldstein, MD, MBA, President and Chief Medical Officer Consistency of Outcomes with Injectable Human Corneal Endothelial Cell Therapy Across Multiple Clinical Trials Monday, September 9, 2024 5:36 p.m. – 5:42 p.m. CET Free paper podium 2, Fira Gran Via, Barcelona Presenter: Eris Jordan, OD, Vice President, Global Clinical & Medical Affairs Aurion Biotech’s drug candidate, AURN001, recently received both Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation from the U.S. Food and Drug Administration for the treatment of corneal edema secondary to corneal endothelial disease. In Japan, Aurion has received both regulatory and reimbursement approval for the treatment of bullous keratopathy, marking the first-ever approval in the world for an allogeneic cell therapy to treat corneal endothelial disease. About Aurion Biotech Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. It received the prestigious Prix Galien award for best start-up in biotech in 2022. Its first product candidate is for the treatment of corneal edema secondary to corneal endothelial disease, and the first clinically validated cell therapy for corneal care, having received regulatory and reimbursement approval in Japan. The Company has completed enrollment and dosing of its Phase 1 / 2 clinical trial in the U.S. and Canada. Privately held, Aurion Biotech is backed by Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures. To learn more, visit www.aurionbiotech.com .

First allogeneic cell therapy to receive both FDA designations for the treatment of corneal edema secondary to corneal endothelial disease SEATTLE & CAMBRIDGE, Mass. & TOKYO--(BUSINESS WIRE)-- Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation for AURN001, the Company’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease. Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). RMAT is part of the 21st Century Cures Act1. An investigational drug is eligible for RMAT designation if: it meets the definition of regenerative medicine therapy; is intended to treat, modify, reverse, or cure a serious condition; and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such conditions. Sponsor companies receiving BTD and RMAT Designation can benefit from increased interactions with the FDA with the goal of expediting drug development. The dual designations granted to Aurion Biotech are based on the criteria stated above, the unmet medical need among patients with corneal endothelial disease, and FDA’s review of clinical data from the AURN001 program, which includes multiple clinical trials with subjects having completed at least 12 months of follow-up. “Aurion Biotech is honored to receive the Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for AURN001,” said Sterling Chung, Vice President of Regulatory Affairs and Quality at Aurion Biotech. “These designations underscore the importance of developing a potential solution for millions of patients around the world who suffer from corneal endothelial diseases. We look forward to working closely with the FDA to expedite the development of our cell therapy.” This news comes on the heels of Aurion’s announcement that it has completed enrollment and dosing of its Phase 1 / 2 CLARA trial in the U.S. and Canada2. The CLARA clinical trial is a prospective, multi-center, randomized, double-masked, parallel-arm cell dose-ranging study in subjects with corneal edema secondary to corneal endothelial dysfunction. The study will evaluate three different doses of neltependocel used in combination with Y-27632. Ninety-seven subjects have been randomized at US & Canadian sites. The CLARA trial is designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction. The primary endpoint is the percentage of subjects who gain three (3) lines of vision at six (6) months. The company has already received regulatory approval in Japan. “The BTD and RMAT designations are important milestones for people living with corneal endothelial disease who need better, less burdensome, more accessible treatment options,” said Greg Kunst, chief executive officer of Aurion. “I thank the Aurion team for their hard work and dedication, and I’m grateful for the essential contributions of clinical trial patients and their ophthalmologists - all of whom have helped to make today’s news a reality.” About Corneal Endothelial Dysfunction Corneal edema secondary to endothelial dysfunction is a sight-threatening and debilitating condition affecting millions of people throughout the world. When corneal endothelial cells die or degrade, they do not regenerate. If left untreated, corneal endothelial cell loss can cause corneal edema (swelling) and loss of vision. Although corneal transplants are effective, there are disadvantages with these procedures – PKP/DMEK/DSAEK – including limited donor organ supply. Transplants require a supply of donor corneas in a 1:1 ratio (one healthy donor cornea to treat each diseased one), yet it’s estimated that there is only one donor cornea available for every 70 diseased eyes. In addition, post-operative recovery for corneal transplant patients requires that they lie flat on their backs for up to three days, in order for the transplant to adhere to the corneal stroma. About Aurion Biotech Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. It received the prestigious Prix Galien award for best start-up in biotech. Its first candidate is for the treatment of corneal edema secondary to corneal endothelial disease, and the first clinically validated cell therapy for corneal care, having received regulatory approval in Japan. The Company has completed enrollment and dosing of its Phase 1 / 2 clinical trial in the U.S. and Canada. Privately held, Aurion Biotech is backed by Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures. To learn more, visit 1 2

COLUMBUS, Ohio, May 23, 2024 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected to manufacture adeno-associated vectors (AAV) therapies using its suspension AAV Curator™ Platform under the California Institute for Regenerative Medicine (CIRM) Accelerating Medicines Partnership (AMP) Bespoke Gene Therapy Consortium (BGTC). Continue Reading The CIRM partnership with Andelyn was established to advance BGTC's mission to develop platforms and standards to propel the development and delivery of customized or 'bespoke' gene therapies for millions of people affected with rare diseases. BGTC is focusing on development of cures for eight such diseases, with Andelyn selected to optimize and scale the AAV therapy processes for the treatment of Congenital Hereditary Endothelial Dystrophy – Type 1. Andelyn Biosciences to manufacture gene therapies using its suspension AAV Curator(TM) Platform for CIRM Post this Matt Niloff, Chief Commercial Officer of Andelyn, said: "As a long-standing pioneer in AAV gene therapies and historical connection to the clinic, we have great synergies with the CIRM/BGTC. Andelyn is privileged to work with the CIRM/BGTC on its first AMP established specifically for rare disease and leverage our AAV Curator™ Platform to help overcome the major obstacles related to developing gene therapies." Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM said: "This partnership with Andelyn will enable progress in the field and help overcome manufacturing obstacles that can potentially lead to breakthroughs for rare diseases like Congenital Hereditary Endothelial Dystrophy." With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases. About Andelyn Biosciences, Inc. Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn can accelerate programs and deliver high quality products by developing and manufacturing processes on its configurable, data-driven AAV Curator™ Platform, or tech transfer in an established client program. Capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A rigorous quality system, regulatory support and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit andelynbio.com. About the California Institute for Regenerative Medicine (CIRM) At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to SOURCE Andelyn Biosciences